BIO appreciates the opportunity to comment on these proposed revisions. Patients living with rare diseases often experience significant unmet medical need due to the lack of knowledge about how these diseases are caused or inherited and their progression. That those suffering from rare diseases are predominately children raises issues around how society prioritizes and develops treatments for these conditions. We believe there are significant challenges in reconciling existing population-level value assessment methodologies with the varied healthcare contexts and deeply personal patient-level treatment decisions faced by patients afflicted with rare diseases, their families, and their clinicians. For a number of reasons, applying a patient-centric lens when considering the value of treatment is especially important when considering rare diseases.

Dear Dr. Pearson:

We are writing on behalf of the Biotechnology Innovation Organization (BIO) to provide comments on the Institute for Clinical and Economic Review’s (ICER) "Proposed adaptation of the ICER value framework for the assessment of treatments for ultra-rare conditions" (modified Framework).1 BIO is the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology companies, state biotechnology centers, and related organizations across the United States and in more than 30 other nations. BIO’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place. In that way, our members’ novel therapeutics, vaccines, and diagnostics not only have improved health outcomes, but have also reduced healthcare expenditures due to fewer physician office visits, hospitalizations, and surgical interventions.

BIO appreciates the opportunity to comment on these proposed revisions. Patients living with rare diseases often experience significant unmet medical need due to the lack of knowledge about how these diseases are caused or inherited and their progression. That those suffering from rare diseases are predominately children raises issues around how society prioritizes and develops treatments for these conditions. We believe there are significant challenges in reconciling existing population-level value assessment methodologies with the varied healthcare contexts and deeply personal patient-level treatment decisions faced by patients afflicted with rare diseases, their families, and their clinicians. For a number of reasons, applying a patient-centric lens when considering the value of treatment is especially important when considering rare diseases:

The health consequences of rare diseases can often be debilitating or deadly;

The manifestation of the burden for a given disease is often unique to individual patients and their caregivers; and

The healthcare needs of patients with rare diseases are underrepresented in healthcare policy discussions and treatment delivery systems, which are typically focused on broader population healthcare.

Given these factors, we strongly believe that the application of a population-based approach (such as the cost per quality-adjusted life year, or QALY) to value treatment for rare diseases is fundamentally misguided because the assessment principles of population-based approaches inherently under-value the unique considerations appropriate for rare diseases. As such, we believe that a new and innovative approach to value rare diseases should be created; one that focuses wholly on the value of innovation to the individual, their caregivers, and society.